Ontology highlight
ABSTRACT:
SUBMITTER: Yiu WH
PROVIDER: S-EPMC2889730 | biostudies-literature | 2010 Jun
REPOSITORIES: biostudies-literature
Yiu Wai Han WH Lee Young Mok YM Peng Wen-Tao WT Pan Chi-Jiunn CJ Mead Paul A PA Mansfield Brian C BC Chou Janice Y JY
Molecular therapy : the journal of the American Society of Gene Therapy 20100413 6
Glycogen storage disease type Ia (GSD-Ia) patients deficient in glucose-6-phosphatase-alpha (G6Pase-alpha or G6PC) manifest disturbed glucose homeostasis. We examined the efficacy of liver G6Pase-alpha delivery mediated by AAV-GPE, an adeno-associated virus (AAV) serotype 8 vector expressing human G6Pase-alpha directed by the human G6PC promoter/enhancer (GPE), and compared it to AAV-CBA, that directed murine G6Pase-alpha expression using a hybrid chicken beta-actin (CBA) promoter/cytomegaloviru ...[more]